Newsletter

Dear CTNNB1 community, As we prepare to gather once again for our annual conference, this month’s newsletter is filled with exciting updates from across our global community. From major research milestones and clinical trial progress to preparations for the upcoming conference in Barcelona, there is much to celebrate and look forward to. Inside, you’ll find highlights from recent […]

Dear CTNNB1 community, This month’s newsletter brings several important milestones and updates from across the CTNNB1 community. We share exciting progress from the GAIN clinical trial, including the successful treatment of the second patient in the world with our investigational gene therapy URBAGEN intended to treat CTNNB1 syndrome.  We also reflect on recent community activities, from the ESGCT Summer School in Coimbra

Dear CTNNB1 community, We took a short break at the beginning of the year due to an exceptionally busy period, but we’re excited to be back with a new edition of our monthly newsletter. 🗞️ This issue brings both hope and honesty. 💙 We’re pleased to share key advancements in our gene therapy program, including

Dear CTNNB1 community, As we enter the heart of the holiday season, we hope you’re able to slow down, find moments of warmth, and enjoy time with the people you love. This is a busy and meaningful month for our community, and we are grateful for every family, researcher, clinician, and supporter who continues to

Dear CTNNB1 community, This month marks a truly extraordinary moment in our shared journey – the approval of our first-ever clinical trial for CTNNB1 syndrome. Together, we’ve turned what once felt like a distant dream into a concrete reality. Every late night, every donation, every show of support has led us to this point. In

Dear CTNNB1 community, As summer comes to a close, we hope you’ve had time to rest, recharge, and enjoy moments with your loved ones. Many of our CTNNB1 children are also heading back to school, and we wish them lots of success, growth, and – most importantly – fun in the year ahead. 🎒🍀 With

Dear CTNNB1 community, We took a little break from the newsletter last month and we’ve missed connecting with you! Can you believe summer is already halfway through? We hope you’ve had a chance to relax, recharge, or still have a vacation to look forward to. ☀️🏖️ While you’ve (hopefully) been soaking up some sunshine, our

Dear CTNNB1 community, Time is flying – we’re just two weeks away from the CTNNB1 Conference in Bilbao, and we can’t wait to welcome you there! In this month’s newsletter, you’ll find updates on what we’ve been working on, along with exciting new resources and an inspiring personal story about growing up with CTNNB1 syndrome.

Dear CTNNB1 community, This month has been a whirlwind of looming deadlines and promising milestones—most excitingly, we’re edging ever closer to the start of clinical trials. Knowing the finish line is in sight fuels us every day. We hope you enjoyed restorative holidays with loved ones and are ready for what’s next. With the CTNNB1 Conference just

We’re proud to launch the very first edition of our Monthly Newsletter! 🎉 This marks a new chapter in how we connect as a community. Each month, we’ll be sharing updates from the CTNNB1 Foundation, progress in research, inspiring stories from families, expert insights, and opportunities to get involved. In this issue: Community news &